TW202321449A - 用於寡核苷酸劑的基於寡核苷酸的遞送載體及其使用方法 - Google Patents
用於寡核苷酸劑的基於寡核苷酸的遞送載體及其使用方法 Download PDFInfo
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- TW202321449A TW202321449A TW111125375A TW111125375A TW202321449A TW 202321449 A TW202321449 A TW 202321449A TW 111125375 A TW111125375 A TW 111125375A TW 111125375 A TW111125375 A TW 111125375A TW 202321449 A TW202321449 A TW 202321449A
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
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- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN2021105081 | 2021-07-07 | ||
| WOPCT/CN2021/105081 | 2021-07-07 | ||
| WOPCT/CN2022/091076 | 2022-05-06 | ||
| CN2022091076 | 2022-05-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW202321449A true TW202321449A (zh) | 2023-06-01 |
Family
ID=84801315
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW111125375A TW202321449A (zh) | 2021-07-07 | 2022-07-06 | 用於寡核苷酸劑的基於寡核苷酸的遞送載體及其使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20250297258A1 (https=) |
| EP (1) | EP4367244A4 (https=) |
| JP (1) | JP2024523702A (https=) |
| KR (1) | KR20240043822A (https=) |
| CN (1) | CN117916374A (https=) |
| AU (1) | AU2022308123A1 (https=) |
| CA (1) | CA3225998A1 (https=) |
| TW (1) | TW202321449A (https=) |
| WO (1) | WO2023280190A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2025522433A (ja) | 2022-06-15 | 2025-07-15 | アローヘッド ファーマシューティカルズ インコーポレイテッド | スーパーオキシドジスムターゼ1(SOD1)の発現を阻害するためのRNAi剤、その組成物、及び使用の方法 |
| KR20250148653A (ko) * | 2023-02-17 | 2025-10-14 | 아이오니스 파마수티컬즈, 인코포레이티드 | App 발현을 감소시키기 위한 화합물 및 방법 |
| KR20250155023A (ko) * | 2023-02-24 | 2025-10-29 | 락티젠 세러퓨틱스 | Sod1을 표적으로 하는 올리고뉴클레오타이드 |
| WO2024199445A1 (en) * | 2023-03-30 | 2024-10-03 | Ractigen Therapeutics | Vehicles for delivering oligonucleotides and methods of use thereof |
| EP4728073A1 (en) * | 2023-06-16 | 2026-04-22 | Ractigen Therapeutics | Oligonucleotide formulation |
| CN117402096A (zh) * | 2023-10-20 | 2024-01-16 | 康羽生命科学技术(苏州)有限公司 | 一种辅氨酸衍生物的合成方法 |
| WO2025119303A1 (en) * | 2023-12-07 | 2025-06-12 | Ractigen Therapeutics | Abasic modification compound, gene modulating agents and the use thereof |
| WO2025126153A2 (en) * | 2023-12-14 | 2025-06-19 | Aviadobio Ltd. | Compositions and methods for treating sod1-mediated neurological diseases |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2715289C (en) * | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | Modified rnai polynucleotides and uses thereof |
| EP2342340A1 (en) * | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| EP2362729A1 (en) * | 2008-11-17 | 2011-09-07 | Enzon Pharmaceuticals, Inc. | Releasable conjugates for nucleic acids delivery systems |
| US9745574B2 (en) * | 2009-02-04 | 2017-08-29 | Rxi Pharmaceuticals Corporation | RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| EP2756845B1 (en) * | 2009-04-03 | 2017-03-15 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA |
| RU2615143C2 (ru) * | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| CA2871089A1 (en) * | 2011-04-20 | 2012-10-26 | Smith Holdings, Llc | Methods and compositions for modulating gene expression using components that self assemble in cells and produce rnai activity |
| EP3004347B1 (en) * | 2013-05-30 | 2018-09-26 | National University Corporation Tokyo Medical and Dental University | Double-stranded agents for delivering therapeutic oligonucleotides |
| EP3862005A1 (en) * | 2015-07-06 | 2021-08-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (sod1) |
| US11260134B2 (en) * | 2016-09-29 | 2022-03-01 | National University Corporation Tokyo Medical And Dental University | Double-stranded nucleic acid complex having overhang |
| SG10201609048RA (en) * | 2016-10-28 | 2018-05-30 | Agency Science Tech & Res | Antisense oligonucleotides |
| US20210002640A1 (en) * | 2018-02-28 | 2021-01-07 | Exicure, Inc. | Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors |
| CN113614232A (zh) * | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
-
2022
- 2022-04-18 US US18/577,092 patent/US20250297258A1/en active Pending
- 2022-07-06 CN CN202280048479.9A patent/CN117916374A/zh active Pending
- 2022-07-06 AU AU2022308123A patent/AU2022308123A1/en active Pending
- 2022-07-06 KR KR1020247004696A patent/KR20240043822A/ko active Pending
- 2022-07-06 CA CA3225998A patent/CA3225998A1/en active Pending
- 2022-07-06 WO PCT/CN2022/104037 patent/WO2023280190A1/en not_active Ceased
- 2022-07-06 EP EP22836938.5A patent/EP4367244A4/en active Pending
- 2022-07-06 JP JP2024500176A patent/JP2024523702A/ja active Pending
- 2022-07-06 TW TW111125375A patent/TW202321449A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20250297258A1 (en) | 2025-09-25 |
| AU2022308123A1 (en) | 2024-02-22 |
| JP2024523702A (ja) | 2024-06-28 |
| EP4367244A4 (en) | 2025-07-09 |
| CA3225998A1 (en) | 2023-01-12 |
| EP4367244A1 (en) | 2024-05-15 |
| KR20240043822A (ko) | 2024-04-03 |
| CN117916374A (zh) | 2024-04-19 |
| WO2023280190A1 (en) | 2023-01-12 |
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